Mi­cro­cap Capri­cor soars on in­ter­im PhII DMD da­ta show­ing func­tion­al ben­e­fit for old­er pa­tients

With the in­ter­im analy­sis for its Phase II Duchenne mus­cu­lar dy­s­tro­phy, lit­tle Capri­cor Ther­a­peu­tics was mere­ly look­ing for a guide­post, a sig­nal, a trend that the ef­fects seen in an ear­li­er tri­al can be repli­cat­ed with a new dos­ing reg­i­men and de­liv­ery method. In­stead, the biotech found it­self blessed with a bas­ket of da­ta points that seem to buck the nat­ur­al his­to­ry trend and sep­a­rate the drug from place­bo in a sta­tis­ti­cal­ly sig­nif­i­cant way, more than dou­bling its tiny stock $CAPR in pre-mar­ket trad­ing.

Lin­da Mar­bán Capri­cor

The pri­ma­ry end­point for the HOPE-2 tri­al is PUL 2.0, a mea­sure of up­per limb func­tion and one of sev­er­al ways Capri­cor quan­ti­fied skele­tal mus­cle im­prove­ment. Ex­ecs al­so spot­light­ed pul­monary and car­diac ben­e­fits in the top-line re­sults — fac­tors that are par­tic­u­lar­ly for the pa­tient pop­u­la­tion that they are tar­get­ing, who tend to be old­er and non-am­bu­la­to­ry.

Con­sist­ing of prog­en­i­tor cells de­rived from donor hearts, CAP-1002 is de­signed to re­lease ex­o­somes that kick off a cy­cle of mus­cle re­pair by sup­press­ing in­flam­ma­tion and dri­ving im­munomod­u­la­tion.

Cur­rent­ly, a num­ber of DMD pa­tients take glu­co­cor­ti­cos­teroids for that pur­pose, in­clud­ing pred­nisone and de­flaza­cort (sold in the US by Marathon as Em­flaza). There’s al­so Sarep­ta’s ex­on-skip­ping drug, eteplirsen (Ex­ondys 51), though its ben­e­fits have yet to be borne out; Sarep­ta and a hand­ful of ri­vals are now in hot pur­suit of a sup­posed one-time cure to re­place the miss­ing dy­s­trophin gene in pa­tients.

“While there had been ad­vances in gene ther­a­py, we be­lieve that con­trol­ling in­flam­ma­tion over and above what can be done by steroids is im­por­tant,” CEO Lin­da Mar­bán said in a con­fer­ence call.

Her hus­band, Ed­uar­do Mar­bán, had de­vel­oped the tech at Johns Hop­kins.

Da­ta from the small, place­bo-con­trolled study sug­gest that with a high­er dose than the past and de­liv­ered in­tra­venous­ly, the ther­a­py is work­ing. At the six-month cut, 6 pa­tients were evalu­able in each arm. For those treat­ed with CAP1002 mid-lev­el PUL — think mov­ing el­bows and pulling hand to mouth — de­te­ri­o­rat­ed by an av­er­age of 0.2 on a 6-point scale, com­pared to 0.8 on place­bo. The p-val­ue was 0.0389.

This and grip strength are the on­ly im­prove­ments still sta­tis­ti­cal­ly sig­nif­i­cant af­ter six months (there was al­so a three-month snap­shot), but Capri­cor stressed pos­i­tive trends through­out.

“I’d like to just re­mind peo­ple that this pop­u­la­tion of DMD re­al­ly rep­re­sents more than half of all DMD pa­tients and there re­al­ly have not been ther­a­pies that have re­al­ly fo­cused on this pop­u­la­tion which have just in­cred­i­bly high bur­den of dis­ease,” Craig Mc­Don­ald, the na­tion­al PI and a pro­fes­sor at UC Davis, said in the call.

On the key is­sue of safe­ty, Capri­cor not­ed that there had on­ly been one se­ri­ous ad­verse event among 30 in­fu­sions af­ter it put a stan­dard pre-med­ica­tion reg­i­men in place to mit­i­gate po­ten­tial im­mune re­ac­tions. Two pa­tients had had im­me­di­ate im­mune re­ac­tions to the treat­ment late last year, which prompt­ed the com­pa­ny to put a vol­un­tary hold on the tri­al.

Armed with these num­bers and an RMAT des­ig­na­tion from the FDA, Capri­cor is now look­ing in­to a “lean, ef­fi­cient” piv­otal study to po­si­tion the cell ther­a­py for a BLA — if it can find the cash to do so.

A few months ago it was forced to chop 21 staffers, or half of its work­force, in or­der to car­ry on with clin­i­cal ac­tiv­i­ties un­til the end of this year.

So­cial im­age: Shut­ter­stock

Am­gen lays off about 300 work­ers, cit­ing 'in­dus­try head­wind­s'

Amgen has laid off about 300 employees, a company spokesperson confirmed to Endpoints News via email Sunday night.

Employees posted to LinkedIn in recent days about layoffs hitting Amgen last week. The Thousand Oaks, CA-based biopharma, which employs about 24,000 people, said the reduction “mainly” impacted US-based workers on its commercial team.

Drug developers of all sizes, including small upstarts and pharma giants, have let employees go in recent months as the biopharma market drags through a quarters-long winter doldrum.

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New York City in­vests $20M in­to biotech 'in­no­va­tion space' at the Brook­lyn Navy Yard

New York City is investing $20 million in biotech this year in the form of a 50,000-square-foot “innovation space” at the Brooklyn Navy Yard, complete with offices, research laboratories and events and programming space to grow biotech startups and companies.

Mayor Eric Adams said during his State of The City Address last Thursday that there will be an “emphasis” on making more opportunities for women and people of color to further diversify the industry. The City first reported the news.

Bob Bradway, Amgen CEO (Stephen Lam/Reuters)

Am­gen launch­es the first US Hu­mi­ra biosim­i­lar at two dif­fer­ent list prices

The bizarre dynamics of the US prescription drug market were on full display once again this morning as Amgen announced that it would launch the first US biosimilar for Humira, the best-selling drug of all time, at two completely different list prices.

One price for Amgen’s Amjevita (adalimumab-atto) will be 55% below the current Humira list price, which is about $84,000 per year, and another at a list price 5% below the current Humira list price, but presumably (pharma companies don’t disclose rebates) with high rebates to attract PBMs and payers.

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Boehringer In­gel­heim touts pre­ven­tion re­sults in rarest form of pso­ri­a­sis

Boehringer Ingelheim uncorked some positive results suggesting that Spevigo can help prevent flare-ups in patients with a severe form of psoriasis, months after the drug was approved to treat existing flares.

Spevigo, an IL-36R antibody also known as spesolimab, met its primary and a key secondary endpoint in the Phase IIb EFFISAYIL 2 trial in patients with generalized pustular psoriasis (GPP), Boehringer announced on Monday. While the company is keeping the hard numbers under wraps until later this year, it said in a news release that it anticipates sharing the results with regulators.

Chad Mirkin, Flashpoint co-founder

‘The field is at a flash­point’: New Chad Mirkin-found­ed biotech hopes to make more ef­fec­tive can­cer vac­cines

Following the success of the mRNA Covid vaccines, cancer vaccines are seeing renewed interest after years of middling results. But a group of researchers suggests that more attention needs to be paid not to what goes into those vaccines, but how the parts are put together.

In a recent paper published in Nature Biomedical Engineering, researchers led by Northwestern University’s Chad Mirkin describe how the placement of different antigens in a cancer vaccine impacts its efficacy. The paper builds on past work done by Mirkin’s lab that suggests the structure, or how the parts of a vaccine are arranged, impact a vaccine’s efficacy, not just its components.

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Dirk Thye, Quince Therapeutics CEO

Af­ter piv­ot­ing from Alzheimer's to bone con­di­tions, biotech piv­ots again — and halves its head­count

When troubled public biotech Cortexyme bought a private startup named Novosteo and handed the keys to its executive team, the company — which changed its name to Quince Therapeutics — said it would shift its focus from an unorthodox Alzheimer’s approach to Novosteo’s bone-targeting drug platform.

Less than a year later, Quince is pivoting again.

The biotech has decided to out-license its bone-targeting drug platform and its lead drug, NOV004, and instead look for clinical-stage programs to in-license or acquire, according to a press release.

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As­traZeneca, No­vo Nordisk and Sanofi score 340B-re­lat­ed ap­peals court win over HHS

AstraZeneca, Novo Nordisk, and Sanofi won an appeals court win on Monday, as the US Court of Appeals for the Third Circuit found that the companies cannot be forced to provide 340B-discounted drugs purchased by hospitals from an unlimited number of community and specialty pharmacies.

“Legal duties do not spring from silence,” the decision says as the court makes clear that the federal government’s interpretation of the “supposed requirement” that the 340B program compels drugmakers to supply their discounted drugs to an unlimited number of contract pharmacies is not correct, noting:

Ap­peals court toss­es J&J's con­tro­ver­sial 'Texas two-step' bank­rupt­cy case

A US appeals court has ruled against Johnson & Johnson’s use of bankruptcy to deal with mounting talc lawsuits, deciding that doing so would “create a legal blind spot.”

The Third Circuit Court of Appeals reversed a previous bankruptcy court decision on Monday, calling for the dismissal of a Chapter 11 filing by J&J’s subsidiary LTL Management.

Faced with more than 38,000 lawsuits alleging its talc-based products caused cancer, J&J spun its talc liabilities into a separate company called LTL Management back in October 2021 and filed for bankruptcy, a controversial move colloquially referred to as a “Texas two-step” bankruptcy. Claimants argued that the strategy is a misuse of the US bankruptcy code — and on Monday, a panel of judges agreed.

#JPM23: Reg­u­la­to­ry un­cer­tain­ty? What about M&A? Da­ta rule? Alessan­dro Masel­li and John Car­roll take out their crys­tal balls

Endpoints editor and founder John Carroll sat down the Catalent CEO Alessandro Maselli to talk about what’s ahead in 2023. Right or wrong, this covers all the big issues faced by biopharma. This transcript has been edited for brevity and clarity.

John Carroll:

I think 2022 had to be one of the worst years ever for crystal balls. You went into 2022 thinking all sorts of nice things about what was ahead, not thinking about a European land war, maybe not thinking that the Federal Reserve was going to be jacking up interest rates as fast as they could to get ahead of inflation. Just a tremendous number of macroeconomic issues that were out there. The sudden and complete collapse of support on the markets in Nasdaq for biotech. A lot of darlings in the industry that had been out there for a while suddenly found themselves moving from a really hot market to a really cold market all of a sudden and had to make a lot of different changes in terms of strategizing.

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